TY - JOUR
T1 - International experiences in multicriteria decision analysis (MCDA) for evaluating orphan drugs
T2 - a scoping review
AU - Lasalvia, P.
AU - Prieto-Pinto, L.
AU - Moreno, M.
AU - Castrillón, J.
AU - Romano, G.
AU - Garzón-Orjuela, N.
AU - Rosselli, D.
N1 - Publisher Copyright:
© 2019, © 2019 Informa UK Limited, trading as Taylor & Francis Group.
PY - 2019/7/4
Y1 - 2019/7/4
N2 - Introduction: Orphan diseases are low-prevalence conditions with chronically debilitating or life-threatening consequences. Their treatments are generally called orphan drugs (OD). Health–technology assessment processes have traditionally considered cost-effectiveness analysis (CEA), when making reimbursement and pricing decisions for health-care plans. Valuing OD with standard CEA raises important issues due to uncertain evidence, inability to meet cost-effectiveness thresholds for reimbursement and high budget impact, among others. Multi-criteria decision analysis (MCDA) allows to overcome these issues and improve the technical and ethical quality of decisions regarding prioritization, coverage, and reimbursement of OD. Areas covered: A scoping review was conducted in order to characterize MCDA frameworks for assessing OD and implementation experiences. We reviewed electronic databases (Medline, Embase, Cochrane Library, EBSCO, CINAHL, EconLit, Web of Science, LILACS, Google Scholar) key journals (Orphanet Journal of Rare Diseases and Value in Health) and organization repositories. Expert opinion: The theoretical framework for MCDA considers areas related to characteristics of orphan diseases and their technologies’ clinical and economic impact. Participation processes are critical in incorporating societal values in weighting different dimensions and constructing decision rules. Local implementation pilots considering different stakeholders are necessary in order to pinpoint specific barriers and opportunities.
AB - Introduction: Orphan diseases are low-prevalence conditions with chronically debilitating or life-threatening consequences. Their treatments are generally called orphan drugs (OD). Health–technology assessment processes have traditionally considered cost-effectiveness analysis (CEA), when making reimbursement and pricing decisions for health-care plans. Valuing OD with standard CEA raises important issues due to uncertain evidence, inability to meet cost-effectiveness thresholds for reimbursement and high budget impact, among others. Multi-criteria decision analysis (MCDA) allows to overcome these issues and improve the technical and ethical quality of decisions regarding prioritization, coverage, and reimbursement of OD. Areas covered: A scoping review was conducted in order to characterize MCDA frameworks for assessing OD and implementation experiences. We reviewed electronic databases (Medline, Embase, Cochrane Library, EBSCO, CINAHL, EconLit, Web of Science, LILACS, Google Scholar) key journals (Orphanet Journal of Rare Diseases and Value in Health) and organization repositories. Expert opinion: The theoretical framework for MCDA considers areas related to characteristics of orphan diseases and their technologies’ clinical and economic impact. Participation processes are critical in incorporating societal values in weighting different dimensions and constructing decision rules. Local implementation pilots considering different stakeholders are necessary in order to pinpoint specific barriers and opportunities.
KW - Health technology assessment
KW - MCDA
KW - multi-criteria decision analysis
KW - orphan disease
KW - orphan drugs
KW - rare disease
KW - scoping review
UR - http://www.scopus.com/inward/record.url?scp=85069524482&partnerID=8YFLogxK
U2 - 10.1080/14737167.2019.1633918
DO - 10.1080/14737167.2019.1633918
M3 - Review article
C2 - 31210065
AN - SCOPUS:85069524482
SN - 1473-7167
VL - 19
SP - 409
EP - 420
JO - Expert Review of Pharmacoeconomics and Outcomes Research
JF - Expert Review of Pharmacoeconomics and Outcomes Research
IS - 4
ER -
